News

Look hard before you leap: the use of stem cells for cerebral palsy remains unproven

23 August 2018

For parents whose children have been diagnosed with cerebral palsy, a permanent disability that affects movement and posture for 1 in 500 babies born in Australia, stem cell-based treatments may be seen as a reasonable option – something worth exploring. Media coverage of “miracle” treatments using stem cells, and on-line advertising by commercial clinics, certainly imply that stem cells can already help. However, behind these often-simplistic presentations there are complex underlying issues that should be fully considered and discussed with the paediatrician and healthcare team supporting the family.

What is actually involved and does it pose risk to the child’s health?

Exploring whether stem cell-based treatments could help children with cerebral palsy is still at a very early stage of development and no proven treatments are currently available. Various new strategies using stem cells are being explored to limit the damage to cells in the brain and reduce the symptoms that children experience. However, effectively treating cerebral palsy is challenging because many different types of cells in the brain can be affected.

Researchers are exploring different strategies. Some are looking to use stem cells to replace lost cells, others are examining whether certain stem cells could help protect or repair damaged nerve cells in the brain. Other researchers are exploring whether doctors could use drugs to stimulate the brain to create new cells and repair damaged areas. Research using stem cells in the lab may be helpful in identifying what kinds of drugs could be useful to achieve this goal.

Whilst the researchers are starting to test possible stem cell-based interventions in clinical trials overseas and in Australia, there are currently no clinically approved stem cell treatments for cerebral palsy.

Each of these possible approaches may carry a risk that could harm the child’s health. If cells are not prepared properly, there is a risk that the cells could introduce an infection to the child – even if the patient’s own cells are used. There is also a risk that the cells may not behave as they are expected to. This will be influenced by what type of cells are used and from where they are obtained, as well as what they are asked to do after being injected. For example, putting cells from the bone marrow back into the blood system carries fewer risks as the cell types are the same in both tissues. However, injecting blood or bone marrow cells into the brain or the spinal cord carries a very different risk as such cells don’t belong there and may grow in a way that is not expected.

When it comes to various ‘treatments’ sold by commercial clinics, what is being injected is often impure or undefined. In many instances, the type of stem cell and where they come from isn’t discussed. There may also be no discussion about the processes used to ‘engineer’ the cells and whether this changes the cells and the level of risk of harm for the patient. The methods used to prepare the cells and how they are delivered to the patient is important and should be conducted by experts in accredited laboratories. Just injecting the cells, if not done correctly, can cause harm. A child died in Germany following complications when their own bone marrow stem cells were injected into the brain as a claimed ‘treatment’ for cerebral palsy.

Anecdotal reports are not evidence

It is certainly compelling to read or listen to stories of success where benefit, even minor benefit, is attributed to a particular form of treatment, and it can be hard to ignore. But these stories provide a snapshot in time. It can be unclear if the benefit is long lasting and if the same procedure also helped the next patient. It might be that other treatments also provided at the clinic, such as intensive physiotherapy, could be responsible for the observed improvement.

Only properly conducted clinical trials can gather evidence to fully explore if a proposed treatment is safe and effective for patients with a particular condition. Importantly, evidence gained in clinical trials can identify when it is best to apply a particular procedure to have genuine benefit. Clinical trials provide in-depth care and long-term evaluation and support for the participants in the study. Findings are shared – through publications and presentations at conferences – and scrutinised by other experts to ensure successful new treatments become widely recognised and available to patients around the world.

Clinics selling a treatment are not necessarily performing clinical trials or acting on the evidence of a clinical trial. Indeed, participants in clinical trials are not asked to pay. Anecdotal evidence from a clinic selling a treatment, who have a vested interest in promoting and expanding their business, is not evidence. Conversely, paying for a treatment which proves to be ineffective is likely to remove your ability to be accepted into a future clinical trial.

Consider all aspects about what is being offered and by whom. Discuss what you find in your research with your child’s paediatrician and your healthcare team. It is easy for clinics to claim benefits, but much harder to do recognised and valuable research.